Recruiting

Phase 1

Cell Based TherapyTrial of Cell Based Therapy for DMD

Sponsor:

Masonic Cancer Center, University of Minnesota

Code:

NCT06692426

Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

MyoPAXon

Tacrolimus

Study Details

Brief summary:

This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals with Duchenne muscular dystrophy (DMD)

Conditions

Duchenne Muscular Dystrophy

Study ID

NCT06692426

Start date

Mar 20, 2025

Status verified date

Mar, 2026

Completion date

Mar 3, 2027

Anticipated

Primary completion date

Mar 3, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy
Non-ambulatory
Intact extensor digitorum brevis (EDB) muscles bilaterally
Off investigational therapies for > 30 days
Age 18 years of age or older at the time of consent
Have adequate organ function confirmed by the following laboratory values obtained within 14 days prior to enrollment (28 days for cardiac and pulmonary function):
Participants with partners of childbearing potential must be willing to use at least two forms of effective birth control (one form must be a barrier method) while receiving the study product and for 3 months after stopping tacrolimus therapy.
Ability to follow commands sufficiently to perform voluntary aspects of outcome measures throughout the study period
Willing to consent to monitoring for 15 years, including an extension period, as required for all interventional studies involving the transplantation of cells that have been genetically modified
Voluntary written consent from the subject or parent(s)/guardian(s) and assent from participant prior to the performance of any research related activity.

Exclusion Criteria:

Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
Active treatment with another investigational therapy
Known allergy to MyoPAXon components

Study Design

Enrollment

8 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Arm A: MyoPAXon 25 x 10^6

MyoPAXon will be delivered via open intramuscular (IM) injection into a single EDB muscle for each participant as a one-time dose. The participants will be started on tacrolimus 1 week prior to dosing and stay on the maintenance dose on a BID schedule for 3 months after injection

experimental: Arm B: MyoPAXon 50 x 10^6

experimental: Arm C: MyoPAXon 100 x 10^6

experimental: Arm D: MyoPAXon 200 x 10^6

Interventions

MyoPAXon

MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2

Tacrolimus

Tacrolimus (Prograf) is an immunosuppressant that inhibits calcineurin and T cell activation, and is commonly used to prevent solid organ transplant rejection1 and graft versus host disease (GVHD) as well as allograft rejection in the setting of allogeneic hematopoietic stem cell transplantation.

Primary outcome measure

Maximal tolerable dose (MTD) of MyoPAXon [ Time Frame: 3 months ]

Central Contacts and Locations

Central contacts

Peter Kang, MD

612-624-9452 mdstemcell@umn.edu

Locations

Masonic Cancer Center

Recruiting

Minneapolis, Minnesota, United States, 55455

More Information

Sponsor

Masonic Cancer Center, University of Minnesota

Last update posted

Mar 4, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-29. This information was provided to ClinicalTrials.gov by Masonic Cancer Center, University of Minnesota on 2026-03-04. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.